Lumping rare diseases sharing similar etiologies into broader clinical trials
Noam Y. Harel; originally posted 8/21/2008; updated 3/31/2013
(Inspired by conversations at the Clinical Trials Methods Course sponsored by NINDS and Univ. of Rochester 8/2008; special thanks to Dr. John Marler) (Reinforced by publications that have appeared since originally posting this entry: Dobkin 2009; Hodes et al., 2013)
Rationale:
Many neurological diseases are extremely rare.
Some of these diseases fall into broader groups such as muscular dystrophies, lysosomal storage disorders, or neurodegenerative diseases.
Each of these broad groups of neurological diseases share several putative pathologic mechanisms - for example, protein aggregation in various neurodegenerative diseases; apoptosis in almost all of these diseases.
In many cases, similar drugs are tested in clinical trials for various different diseases in the same broader group - for instance, Coenzyme Q10 in ALS, Parkinson's, and Huntington's disease.
Goal:
To increase recruitment, cut administrative costs, and expand generalizability, conduct larger trials recruiting patients with various diseases within broader groups, with fewer exclusion criteria.
Would need to normalize outcome measures on disease-specific scales - the new NIH Toolbox provides the perfect set of tests to do this.
Use pre-specified subgroup analyses to determine if a drug shows efficacy for one or more of a group of diseases.
Lumping rare diseases sharing similar etiologies into broader clinical trials
Noam Y. Harel; originally posted 8/21/2008; updated 3/31/2013(Inspired by conversations at the Clinical Trials Methods Course sponsored by NINDS and Univ. of Rochester 8/2008; special thanks to Dr. John Marler)
(Reinforced by publications that have appeared since originally posting this entry: Dobkin 2009; Hodes et al., 2013)
Rationale:
Goal: